On January 12, 2022 Castle Biosciences, Inc. (Nasdaq: CSTL), a leader in transforming disease management and improving patient outcomes through innovative diagnostics, reported the publication of a study demonstrating that the combined application of DecisionDx-UM, DecisionDx-PRAME and DecisionDx-UMSeq allows for highly accurate analysis of RNA and DNA from a single biopsy sample for patients with uveal melanoma (UM) (Press release, Castle Biosciences, JAN 12, 2022, View Source [SID1234598635]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

DecisionDx-UMSeq is Castle’s 7-gene test that uses next-generation sequencing (NGS) to identify somatic mutations relevant to uveal melanoma. The sequencing panel identifies hotspot mutations in the genes GNAQ, GNA11, CYSLTR2, PLCB4 and SF3B1, mutations in exons 1-2 of EIF1AX and mutations across all coding exons of the BAP1 gene. This information, together with results from the DecisionDx-UM gene expression profile (GEP) test, is designed to help build a comprehensive genomic profile of an individual UM tumor from a single biopsy, which can then be used to inform patient care. DecisionDx-UM is Castle’s 15-GEP test that uses an individual patient’s tumor biology to predict individual risk of metastasis. DecisionDx-UM is the standard of care in the management of newly diagnosed UM in the majority of ocular oncology practices in the United States.

The study titled, "Analytical validation and performance of a 7-gene next-generation sequencing panel in uveal melanoma," was recently published in the peer-reviewed journal Ocular Oncology and Pathology. The study, which can be viewed here, was designed to evaluate the analytical performance of the DecisionDx-UMSeq panel, which can be run from the same fine needle aspiration biopsy (FNAB) or formalin-fixed paraffin-embedded (FFPE) biopsy sample that is used to run the DecisionDx-UM test.

"The study results demonstrated that running the DecisionDx-UMSeq panel in combination with DecisionDx-UM and DecisionDx-PRAME produced highly accurate results using only a single biopsy sample," said J. William Harbour, M.D., Professor and Chair of the Department of Ophthalmology at UT Southwestern Medical Center. "Patients with the rare and deadly uveal melanoma have limited biopsy tissue available, so it is critical to gather as much molecular information from the tumor as possible to help physicians make the most informed management decisions for their patients."

Study background and highlights:

105 primary tumors were analyzed, including 37 FFPE and 68 FNAB specimens.
DecisionDx-UMSeq achieved a positive percent agreement (PPA) of 100% for detection of both single nucleotide variants (SNVs) and insertions/deletions (INDELs), with a technical positive prediction value (TPPV) of 98.8% and 100%, respectively.
The DecisionDx-UMSeq panel is unique compared to other sequencing panels in that it can be performed from the same biopsy sample that is used to perform the DecisionDx-UM and DecisionDx-PRAME tests and does not require a second biopsy.
Overall, the study data demonstrated that the combined application of DecisionDx-UM, DecisionDx-PRAME and DecisionDx-UMSeq allowed for highly accurate analysis of both RNA and DNA from a single biopsy sample. The ability to perform mutational profiling of UM tumors, in addition to the DecisionDx-UM test, is an important advance that may aid in confirming the presence of a melanocytic lesion, potentially better inform therapy selection, and provide additional layers of prognostic information to help guide patient care.
About DecisionDx-UMSeq

DecisionDx-UMSeq is Castle’s 7-gene DNA sequencing panel that uses NGS to identify somatic mutations relevant to uveal melanoma. The sequencing panel identifies hotspot mutations in the genes GNAQ, GNA11, CYSLTR2, PLCB4 and SF3B1, mutations in exons 1-2 of EIF1AX, and mutations across all coding exons of the BAP1 gene. This information, together with results from the DecisionDx-UM and DecisionDx-PRAME GEP tests, is designed to help build a comprehensive genomic profile of an individual UM tumor from a single biopsy, which can then be used to inform patient care. The DecisionDx-UMSeq DNA sequencing panel is not intended as a substitute for the DecisionDx-UM GEP test. The DecisionDx-UM GEP test is the only uveal melanoma prognostic method that has been prospectively validated in multiple studies, providing the most robust prognostic information currently available for newly diagnosed UM patients to date.

About DecisionDx-UM

DecisionDx-UM is a 15-gene GEP test that uses an individual patient’s tumor biology to predict individual risk of metastasis. DecisionDx-UM is the standard of care in the management of newly diagnosed UM in the majority of ocular oncology practices in the United States. Since 2009, the American Joint Committee on Cancer (AJCC; v7 and v8) Staging Manual for UM has specifically identified this GEP test as a prognostic factor that is recommended for collection as a part of clinical care. Further, the National Comprehensive Cancer Network guidelines for UM include the DecisionDx-UM test result as a prognostic method for determining risk of metastasis and recommended differential surveillance regimens based on a Class 1A, 1B and 2 result. DecisionDx-UM is the only prognostic test for uveal melanoma that has been validated in prospective, multi-center studies, and it has been shown to be a superior predictor of metastasis compared to other prognostic factors, such as chromosome 3 status, mutational status, AJCC stage and cell type.

It is estimated that nearly 8 in 10 patients diagnosed with UM in the U.S. receive the DecisionDx-UM test as part of their diagnostic workup.

About DecisionDx-PRAME

DecisionDx-PRAME is a gene expression profile test that can be run on the same sample used to run DecisionDx-UM to assess expression of the PRAME gene. PRAME, or preferentially expressed antigen in melanoma, is usually not expressed in normal adult tissues, but in some cancers, PRAME expression is elevated. Elevated expression of PRAME has been associated with an increased risk of metastasis in patients with uveal melanoma.

When used in conjunction with results from the DecisionDx-UM test, PRAME expression status may add further precision to the predicted risk of metastasis and help guide physicians and patients to the most appropriate follow-up care regimens.

More information about the tests and disease can be found at www.CastleTestInfo.com.

On January 12, 2022 Hemogenyx Pharmaceuticals plc (LSE: HEMO), the biopharmaceutical group developing new therapies and treatments for blood diseases, and Selexis SA, a JSR Life Sciences company, reported that they have signed a service agreement to develop the cell line for Hemogenyx’s CDX bispecific antibody for the treatment of acute myeloid leukemia (AML) (Press release, Hemogenyx Pharmaceuticals, JAN 12, 2022, View Source [SID1234598634]). Under the agreement, Hemogenyx will leverage Selexis’ proprietary SUREtechnology Platform, a suite of cell line development tools and technologies that significantly reduces the time, effort, and costs associated with developing high-performance mammalian cell lines.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The CDX bispecific is made using the Hemogenyx’s proprietary humanised monoclonal antibody against a target on the surface of AML cells. CDX was co-developed by Hemogenyx Pharmaceuticals and Eli Lilly and Company ("Lilly"). This cutting-edge application of immune therapy offers a potentially more benign and effective form of treatment that, if successful, could have a significant impact on treatment and survival rates for AML. CDX is planned to be the Company’s second therapeutic candidate to enter clinical trials. Following the completion of the co-development phase, Lilly granted the Company an exclusive worldwide license to certain intellectual property developed by Lilly related to the CDX bispecific antibody for all uses, including the treatment of AML and other blood cancers.

Dr. Vladislav Sandler, Chief Executive Officer and co-founder of Hemogenyx Pharmaceuticals commented, "We are delighted to partner with Selexis, and access its proprietary protein expression tools and technologies, IP and know-how. The partnership is key to advancing our CDX programme into clinical trials and accelerating the timeline to deliver this innovative therapy to patients in need of a more benign and effective treatment for AML."

Mr. Dirk Lange, CEO of Selexis, added, "There’s an urgent need for effective treatments for AML, and we at Selexis are pleased to apply our technologies to help Hemogenyx advance the CDX bispecific antibody to the clinic. We’ve built a reputation for delivering cell lines rapidly and cost-effectively, without compromising safety. This is an exciting milestone for Hemogenyx and we welcome the opportunity to join the company on its journey toward delivering a promising and effective therapy for patients with AML."

Selexis’ modular SUREtechnology Platform facilitates the rapid, stable, and cost-effective production of recombinant proteins and vaccines, providing seamless integration of the development continuum from discovery to commercialization.

On January 12, 2022 Kintara Therapeutics, Inc. (Nasdaq: KTRA) ("Kintara" or the "Company"), a biopharmaceutical company focused on the development of new solid tumor cancer therapies, reported that a multiyear research grant from Luxembourg National Research Fund (FNR) and Cancer Foundation Luxembourg has been awarded to Doctor Anna Golebiewska Ph.D., co-leading the NORLUX Neuro-Oncology laboratory with Professor Simone Niclou at the Luxembourg Institute of Health (Press release, Kintara Therapeutics, JAN 12, 2022, View Source [SID1234598633]). The FNR is the main funder of research activities in Luxembourg. The Cancer Foundation Luxembourg is dedicated to patient support and oncology research by providing information to the cancer patient community on preventing, screening and living with the disease. The two organizations jointly fund outstanding projects in the cancer research field. The grant is intended to support Dr. Golebiewska’s research on the mechanism of action for VAL-083’s utility to treat glioblastoma (GBM). Trained as a cellular and molecular biologist, Dr. Golebiewska has been engaged in brain tumor research primarily focused on the biology of GBM with a special interest in tumor heterogeneity, plasticity, and tumor microenvironment.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Along with co-investigator Dr. Petr Nazarov, Ph.D., Dr. Golebiewska’s research project is titled "Deconvolution of heterogeneity in the Glioblastoma cellular ecosystem for understanding treatment resistance and improving patient stratification (DIOMEDES)," and is based on complementary expertise in GBM biology and computational methods for the deconvolution of the complex biological systems associated with the disease. By combining state-of-the art preclinical models, transcriptomic analyses at the single and bulk cell level, and powerful deconvolution methods, the researchers aim to unravel mechanisms that shape treatment resistance in GBM. They will assess transcriptomic changes upon treatment with Temozolomide (TMZ) and VAL-083 in their GBM patient-derived orthotopic xenografts (PDOXs). Direct treatment of PDOXs will allow for the investigation of transcriptomic transitions towards resistant states at the moment of treatment. This research may lead to improved stratification of patients for personalized therapies.

"The recognition and financial support from this esteemed organization to support VAL-083’s research in Dr. Golebiewska’s laboratory is extremely exciting as it will provide important data to optimize the clinical use of this first-in-class small molecule chemotherapeutic," said Dennis Brown, Ph. D., Kintara’s Chief Scientific Officer. "The findings from this research will help elucidate potential therapeutic targets for innovative combination treatment strategies that involve VAL-083 and importantly, further advance the opportunity to increase therapeutic precision when treating GBM patients."

The DIOMEDES project in addition to Kintara’s other scientific collaborations with investigators at M.D. Anderson Cancer Center, University of California San Francisco, and the University of British Columbia, have helped to refine the understanding of VAL-083’s therapeutic potential as a treatment for multiple oncology indications including platinum resistant ovarian cancer, pediatric cancers such as DIPG and Ewings sarcoma, as well as GBM where the utility of the current standard of care (TMZ) is very limited.

On January 12, 2022 KemPharm, Inc. (NasdaqGS: KMPH), a specialty pharmaceutical company focused on the discovery and development of proprietary prodrugs, reported that the Company will host a conference call and live audio webcast on Wednesday, January 19, 2022, at 4:30 p.m. ET, to discuss the Company’s strategy for advancing and expanding its development pipeline. During the call, senior management will provide guidance regarding KemPharm’s future clinical development priorities.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Conference Call Information:

Telephone Access: To access the conference call telephonically, interested participants and investors will be required to register via the following online form: View Source

Once registered, all individuals will be provided with participant dial-in numbers, a passcode and a registrant ID, which can then be used to access the conference call.

Participants may register at any time. It is recommended that the registration process be completed at least 15 minutes prior to the start of the call.

Webcast Access: The live audio webcast with slide presentation will be accessible via the Investor Relations section of KemPharm’s website, View Source An archive of the webcast and presentation will be available for 90 days beginning at approximately 5:30 p.m. ET, on January 19, 2022.

On January 12, 2022 Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX), a clinical stage pharmaceutical company focused on the discovery, development and commercialization of novel therapeutics for rare endocrine diseases and endocrine-related tumors, reported that Scott Struthers, Ph.D., founder & CEO of Crinetics, will provide a company update at the 40th annual J.P. Morgan Healthcare Conference today, Wednesday, January 12th at 4:30 PM Eastern Time / 1:30 PM Pacific Time (Press release, Crinetics Pharmaceuticals, JAN 12, 2022, View Source [SID1234598631]). A live audio webcast of Dr. Struthers’ presentation may be accessed on the Events page of the company’s website.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

During his presentation, Dr. Struthers will discuss Crinetics’ key priorities and anticipated milestones for 2022. These include:

Continued progress in the two ongoing Phase 3 PATHFNDR trials of paltusotine in acromegaly. Both trials remain on track and topline data is expected in 2023.
The initiation of patient dosing in a Phase 2 trial of paltusotine in patients with carcinoid syndrome associated with neuroendocrine tumors (NETs), which is expected in 2022.
Reporting Phase 1 multiple ascending dose (MAD) data for CRN04894, an investigational, oral, nonpeptide adrenocorticotropic hormone (ACTH) antagonist being developed for the treatment of Cushing’s disease and congenital adrenal hyperplasia, which is expected in 1Q 2022.
The initiation of a Phase 2 trial of CRN04894, which is expected in 2H 2022.
Reporting Phase 1 MAD data for CRN04777, an investigational, oral, nonpeptide somatostatin receptor type 5 (SST5) agonist being developed for the treatment of congenital hyperinsulinism, which is expected in 1Q 2022.
The initiation of a Phase 2 trial of CRN04777, which is expected in 2H 2022.
The initiation of IND-enabling studies for a parathyroid receptor type-1 (PTHR1) antagonist, which is expected in 2022. Target indications for this program potentially include hyperparathyroidism and humoral hypercalcemia of malignancy.
"2021 was a transformational year for Crinetics as we achieved a number of important milestones that diversified our clinical pipeline and highlighted the strength of our drug discovery capabilities," stated Dr. Struthers. "We advanced paltusotine into a registrational Phase 3 program and reported Phase 1 clinical proof-of-concept data for both CRN04894 and CRN04777. Each of these data announcements provided additional validation for our drug development roadmap, which aims for early de-risking through animal and healthy volunteer studies leveraging well-established endocrine biomarkers. Looking ahead, we will continue to follow this plan as we work to advance our PTHR1 antagonist program and expand our pipeline. With a talented drug discovery and development team, a steady cadence of catalysts ahead of us, and a strong balance sheet, we believe we are well positioned for sustained success."